The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale; Other: The following data of motor function

• Registration Number: NCT03513367
• Lead Sponsor: University Hospital, Toulouse

Brief Summary

There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy module with a multicentric study. The investigators will evaluate the following psychometric properties : convergent validity, internal validity, inter-rater reliability. The investigators would like to be able to use this scientific tool in future clinical trials.

Detailed Description

The Duchenne Muscular Dystrophy, the commonest form of dystrophy, is an X-linked, recessive neuromuscular disease, in which there is an absence of the protein dystrophin. This chronic and progressive disease leads to an inevitable loss of autonomy (muscle weakness, respiratory and cardiac failure). With better multidisciplinary care, life expectancy has increased but also morbidity. From now one, the evaluation of the quality of life of children with DMD is necessary in therapeutic trials.

Given the specificities of the disease, it seems appropriate to have a specific scale. In the literature there isn't quality of life scale specific to Duchenne Muscular Dystrophy in French version. The only specific scale that exists is the specific module PedsQLTM DMD that was validated in English version in 2012. This scale is relevant for assessing the quality of life in clinical trials and in daily clinical practice given its psychometric properties (good internal consistency close to 0.8).The main hypothesis that we formulate is to validate the French translation of the pediatric module of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale.

Study Timeline

• Study First Submitted: 2018-04-19
• Study First Submitted QC: 2018-04-19
• Study First Posted: 2018-05-01
• Study Start: 2018-09-19
• Status Verified: 2019-04
• Last Update Submitted: 2019-04-16
• Last Update Posted: 2019-04-18
• Primary Completion: 2019-09-19
• Study Completion: 2019-09-19

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 210

Inclusion Criteria

• Boys aged 7 to 18, with genomic Duchenne muscular dystrophy whose parents (mother and / or father) or direct grandparents do not oppose.

Exclusion Criteria

• Inability for the child to understand the issues
• Absence of direct parents or grandparents
• Child receiving antidepressant treatment
• Non French speaking child
• Duchenne Muscular Dystrophy girls

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Boys with Muscular Duchenne Dystrophy	Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale	105 boys with Muscular Duchenne Dystrophy (DMD) distributed as follows: 35 patients with Duchenne muscular dystrophy by age category, 8-12 years old and 13-18 years old. Children will complete the questionnaire of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale regardless of his parents
Parents of boys with Muscular Duchenne Dystrophy	Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale	105 parents of boys with Muscular Duchenne Dystrophy For the 5-7 age group, only parents answer the questionnaire but medical data are collected : 35 by age category (5-7; 8-12; 13-18) Parents will complete the questionnaire of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale regardless of their children
Boys with Muscular Duchenne Dystrophy	The following data of motor function	105 boys with Muscular Duchenne Dystrophy (DMD) distributed as follows: 35 patients with Duchenne muscular dystrophy by age category, 8-12 years old and 13-18 years old. Children will complete the questionnaire of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale regardless of his parents

Primary Outcome Measures

Name	Time	Method
Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale	12 months	The validation process is confirmatory, the scale being widely used in English 201/5000 The internal consistency of the 4 dimensions of the PedsQL ™ DMD module will be evaluated by measuring the Cronbach Alpha. In terms of data availability to children (activity report).; the validation of the DMD module will focus on the validity of constructs, internal structure validity, discriminant validity and reliability
Evaluate the reliability of the French version of the DMD module of the PedsQLTM 3.0 scale	12 months	PedsQLTM is a model for measuring quality of life in children with acute or chronic pathology. Pathology-specific PedsQL ™ provides a better assessment of the quality of life of this population

Trial Locations

Locations (1)

University Hospital Toulouse; 🇫🇷 Toulouse, France